Patients' preferences for treatment outcomes for advanced non-small cell lung cancer: a conjoint analysis.
ABSTRACT Treatment decisions for advanced non-small cell lung cancer (NSCLC) are complex and require trade-offs between the benefits and risks experienced by patients. We evaluated the benefits that patients judged sufficient to compensate for the risks associated with therapy for NSCLC.
Participants with a self-reported diagnosis of NSCLC (n=100) were sampled from an online panel in the United Kingdom. Eligible and consenting participants then completed a self-administered online survey about their disease and their treatment preferences were assessed. This involved respondents choosing among systematically paired profiles that spanned eight attributes: progression-free survival [PFS], symptom severity, rash, diarrhoea, fatigue, nausea and vomiting, fever and infection, and mode of treatment administration (infusion and oral). A choice model was estimated using mixed-logit regression. Estimates of importance for each attribute level and attribute were then calculated and acceptable tradeoffs among attributes were explored.
A total of 89 respondents (73% male) completed all choice tasks appropriately. Increases in PFS together with improvements in symptom severity were judged most important and increased with PFS benefit - 4 months: 5.7; 95% CI: 3.5-7.9; 5 months: 7.1; 95% CI: 4.4-9.9; and 7 months: 10.0; 95% CI: 6.1-13.9. However, improvements in PFS were viewed as most beneficial when disease symptoms were mild and as detrimental when patients had severe symptoms. Fatigue (5.0; 95% CI: 2.7-7.3) was judged to be the most important risk, followed by diarrhoea (2.8; 95% CI: 0.7-4.9), nausea and vomiting (2.1; 95% CI: 0.1-4.1), fever and infection (2.1; 95% CI: 0.2-4.1), and rash (2.0; 95% CI: 0.2-3.9). Oral administration was preferred to infusion (1.8; 95% CI: 0.0-3.6). Patients with mild and moderate symptoms traded PFS for less risks or more convenience if the severe symptoms were not experienced.
This study demonstrates the value of conjoint analysis in the study of patient preferences for cancer treatments. In this small sample of patients with NSCLC from the UK, we demonstrate that the value of improvements in PFS is conditional upon the severity of disease symptoms; and that risks are valued differently.
- [show abstract] [hide abstract]
ABSTRACT: The aim of the study was to quantify patient preferences for outcomes associated with oral antidiabetic medications (OAMs) in Sweden and Germany through a discrete-choice experiment. Adults taking OAMs who had a self-reported physician's diagnosis of type 2 diabetes mellitus (T2DM) made a series of nine choices between pairs of hypothetical profiles. Each profile had a predefined range of attributes: blood glucose control, frequency of mild-to-moderate hypoglycaemia, annual severe hypoglycaemic events, annual weight gain, pill burden and frequency of administration, and cost. Choice questions were based on an experimental design with known statistical properties. Bivariate probit analysis estimated the probabilities of choice of medication administration from patient characteristics and, conditional on that choice, preferences for treatment outcomes. The final sample consisted of 188 Swedish and 195 German patients. For both countries, weight gain was the most important attribute, followed by blood glucose control. Avoiding a 5-kg weight gain was 1.5 times more important in Sweden and 2.3 times more important in Germany than achieving moderate blood glucose control, thereby, suggesting that blood glucose control is relatively more important to Swedish than to German patients. Least important outcomes were the number of daily pills (Sweden) and frequency of mild-to-moderate hypoglycaemia (Germany). Patients in both Sweden and Germany preferred OAMs not associated with weight gain.Diabetes & Metabolism 07/2013; · 2.39 Impact Factor
- [show abstract] [hide abstract]
ABSTRACT: Disease symptom management in patients with advanced non-small cell lung cancer (NSCLC) is a critical aspect of therapy. The main objective of our study was to assess patient-reported outcomes and the degree of concordance between physician and patient perceptions of symptom severity in advanced NSCLC in the USA. Patients with advanced (stage IIIB/IV) NSCLC (N = 450) were recruited in a nationwide (USA) lung cancer study. Patients and their oncologists completed patient and physician versions of the Lung Cancer Symptom Scale (LCSS). Patient-reported lung cancer-specific quality of life was assessed with the Functional Assessment of Cancer Therapy-Lung (FACT-L). Concordance was assessed using the kappa-statistic. Regression analysis was performed with FACT-L total score as the dependent variable and patient-reported LCSS symptom scores as predictors. A high proportion of patients experienced lung cancer symptoms: fatigue (100 %), loss of appetite (97 %), shortness of breath (95 %), cough (93 %), pain (92 %), and blood in sputum (63 %). Concordance between physician and patients was lowest for loss of appetite (kappa 0.1701) and greatest for hemoptysis (kappa 0.4586). Loss of appetite (β = -0.204; p < 0.001), cough (β = -0.145; p < 0.01), pain (β = -0.265; p < 0.001), and shortness of breath (β = -0.145; p < 0.01) were found to be significant predictors of the quality of life. Symptom burden in patients with advanced NSCLC is high and has a negative impact on the quality of life. Patient-reported outcomes data could help optimize disease outcomes and therapy management in NSCLC.Supportive Care in Cancer 09/2013; · 2.09 Impact Factor
- [show abstract] [hide abstract]
ABSTRACT: Decisions regarding the development, regulation, sale, and utilization of pharmaceutical and medical interventions require an evaluation of the balance between benefits and risks. Such evaluations are subject to two fundamental challenges-measuring the clinical effectiveness and harms associated with the treatment, and determining the relative importance of these different types of outcomes. In some ways, determining the willingness to accept treatment-related risks in exchange for treatment benefits is the greater challenge because it involves the individual subjective judgments of many decision makers, and these decision makers may draw different conclusions about the optimal balance between benefits and risks. In response to increasing demand for benefit-risk evaluations, researchers have applied a variety of existing welfare-theoretic preference methods for quantifying the tradeoffs decision makers are willing to accept among expected clinical benefits and risks. The methods used to elicit benefit-risk preferences have evolved from different theoretical backgrounds. To provide some structure to the literature that accommodates the range of approaches, we begin by describing a welfare-theoretic conceptual framework underlying the measurement of benefit-risk preferences in pharmaceutical and medical treatment decisions. We then review the major benefit-risk preference-elicitation methods in the empirical literature and provide a brief overview of the studies using each of these methods. The benefit-risk preference methods described in this overview fall into two broad categories: direct-elicitation methods and conjoint analysis. Rating scales (6 studies), threshold techniques (9 studies), and standard gamble (2 studies) are examples of direct elicitation methods. Conjoint analysis studies are categorized by the question format used in the study, including ranking (1 study), graded pairs (1 study), and discrete choice (21 studies). The number of studies reviewed here demonstrates that this body of research already is substantial, and it appears that the number of benefit-risk preference studies in the literature will continue to increase. In addition, benefit-risk preference-elicitation methods have been applied to a variety of healthcare decisions and medical interventions, including pharmaceuticals, medical devices, surgical and medical procedures, and diagnostics, as well as resource-allocation decisions such as facility placement. While preference-elicitation approaches may differ across studies, all of the studies described in this review can be used to provide quantitative measures of the tradeoffs patients and other decision makers are willing to make between benefits and risks of medical interventions. Eliciting and quantifying the preferences of decision makers allows for a formal, evidence-based consideration of decision-makers' values that currently is lacking in regulatory decision making. Future research in this area should focus on two primary issues-developing best-practice standards for preference-elicitation studies and developing methods for combining stated preferences and clinical data in a manner that is both understandable and useful to regulatory agencies.Applied Health Economics and Health Policy 05/2013;